NHS Approves CRISPR-Based Gene Therapy for Sickle Cell Disease

The UK's National Institute for Health and Care Excellence (NICE) has approved the use of the gene-editing therapy exagamglogene autotemcel (exa-cel) for patients aged 12 and older with severe sickle cell disease, marking a significant advancement in treatment options. This therapy, which utilizes CRISPR technology, was previously rejected by NICE in March 2024 but has now been granted access under a managed access scheme following a commercial agreement with Vertex Pharmaceuticals. The treatment, priced at £1.65 million, allows for the editing of a patient's own stem cells to produce healthy red blood cells, providing a hopeful alternative to donor stem cell transplants, which are limited by donor availability. Campaigners and patients have reacted positively to the news, as around 1,700 individuals could be eligible, with an estimated 50 receiving treatment annually. Clinical trials have shown that exa-cel may effectively halt painful sickle cell crises, offering a potential cure for a condition that severely affects the lives of many. This approval highlights a broader shift towards innovative gene therapies in the treatment of rare genetic disorders.